OBJECTIVES

There is increasing recognition that conventional appraisal approaches (e.g. cost/QALY) may be unsuitable for rare disease treatments. Work Package 10 of the H2020 IMPACT-HTA project aims to develop guidance for an appraisal framework for rare disease treatments. The first task was to document existing HTA appraisal/reimbursement processes in European Member and EEA States, Canada, New Zealand and Australia for rare disease treatments.

METHODS :

Documentation of processes was done through (a) questionnaires to country experts, (b) desk research to complement information received, (c) creation of country vignettes outlining processes and, (d) validation of vignettes and results through follow-up emails or interviews. Case studies were developed to illustrate the impact of the special processes for rare disease treatment.

RESULTS

Eighty four percent (31/37) of countries agreed to participate. 14/31 countries distinguish rare disease and 8/31 ultra-rare disease treatments. 8 countries have special processes using these distinctions: 2 countries account for additional criteria (Sweden, Norway), one implemented an add-on process to the standard process (Scotland), and 5 have distinct processes (England, Germany, Lithuania, Romania, Slovakia). These special processes are characterised by: consideration of additional criteria, more leniency around quality of evidence, special reimbursement status, different requirements for economic models, greater disease-specific expert input, and/or special rare disease appraisal committees. Feedback on their impact were that they resulted in more rare disease treatments being reimbursed and for more nuanced reimbursement decisions. Challenges still remain, e.g. lack of good quality data, interpretation of qualitative data alongside quantitative data, accommodating high prices and ICERs.

CONCLUSIONS :

Many of the processes for appraising drugs for rare diseases are fairly new, allowing countries to gain experience with each new drug appraised and to refine their processes. Characterising them and better understanding their impact and challenges will help further the debate around whether a different appraisal framework is needed for rare disease treatments.